CA3099930A1 - Modified rna agents with reduced off-target effect - Google Patents
Modified rna agents with reduced off-target effect Download PDFInfo
- Publication number
- CA3099930A1 CA3099930A1 CA3099930A CA3099930A CA3099930A1 CA 3099930 A1 CA3099930 A1 CA 3099930A1 CA 3099930 A CA3099930 A CA 3099930A CA 3099930 A CA3099930 A CA 3099930A CA 3099930 A1 CA3099930 A1 CA 3099930A1
- Authority
- CA
- Canada
- Prior art keywords
- muna
- gna
- dsrna
- antisense strand
- thiouridine
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862672405P | 2018-05-16 | 2018-05-16 | |
| US62/672,405 | 2018-05-16 | ||
| US201862719291P | 2018-08-17 | 2018-08-17 | |
| US62/719,291 | 2018-08-17 | ||
| PCT/US2019/032633 WO2019222479A1 (en) | 2018-05-16 | 2019-05-16 | Modified rna agents with reduced off-target effect |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3099930A1 true CA3099930A1 (en) | 2019-11-21 |
Family
ID=68540971
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3099930A Pending CA3099930A1 (en) | 2018-05-16 | 2019-05-16 | Modified rna agents with reduced off-target effect |
Country Status (6)
| Country | Link |
|---|---|
| US (3) | US12247203B2 (https=) |
| EP (1) | EP3802827A4 (https=) |
| JP (3) | JP7384833B2 (https=) |
| AU (2) | AU2019270152B2 (https=) |
| CA (1) | CA3099930A1 (https=) |
| WO (1) | WO2019222479A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019006455A1 (en) | 2017-06-30 | 2019-01-03 | Solstice Biologics, Ltd. | AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF |
| WO2019217459A1 (en) | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| WO2021178607A1 (en) * | 2020-03-05 | 2021-09-10 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases |
| TW202214858A (zh) * | 2020-08-04 | 2022-04-16 | 大陸商上海拓界生物醫藥科技有限公司 | 脫靶活性降低的修飾siRNA |
| WO2022174000A2 (en) | 2021-02-12 | 2022-08-18 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| CN113073098B (zh) * | 2021-02-20 | 2024-08-20 | 北京理工大学 | 抑制BACE1基因表达的siRNA修饰物及其应用 |
| WO2022212231A2 (en) | 2021-03-29 | 2022-10-06 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| KR20240067943A (ko) | 2021-09-24 | 2024-05-17 | 알닐람 파마슈티칼스 인코포레이티드 | 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법 |
| EP4493570A2 (en) | 2022-03-16 | 2025-01-22 | Empirico Inc. | Galnac compositions for improving sirna bioavailability |
| KR20240163743A (ko) | 2022-03-28 | 2024-11-19 | 엠피리코 인크. | 변형된 올리고뉴클레오티드 |
| JP2025524135A (ja) * | 2022-07-27 | 2025-07-25 | イー－セラピューティクス・ピーエルシー | 核酸化合物 |
| JP2025532985A (ja) * | 2022-09-30 | 2025-10-03 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | 修飾二本鎖ｒｎａ剤 |
| WO2024217152A2 (zh) * | 2023-04-21 | 2024-10-24 | 苏州时安生物技术有限公司 | 一种寡聚核酸及其制备方法和应用 |
| WO2025064821A2 (en) | 2023-09-21 | 2025-03-27 | Ionis Pharmaceuticals, Inc. | Compounds and methods for inhibiting lpa |
| US12454693B1 (en) * | 2024-09-27 | 2025-10-28 | Dexcel Pharma Technologies Ltd. | Nucleic acids for inhibiting expression of transferrin receptor 2 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050032069A1 (en) * | 2002-11-05 | 2005-02-10 | Muthiah Manoharan | Oligomeric compounds having modified bases for binding to adenine and guanine and their use in gene modulation |
| US7812149B2 (en) * | 1996-06-06 | 2010-10-12 | Isis Pharmaceuticals, Inc. | 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations |
| EP2152877A2 (en) | 2007-05-02 | 2010-02-17 | Sirna Therapeutics Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE ( PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| JP2012528596A (ja) | 2009-06-03 | 2012-11-15 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | ペプチドダイサー基質剤およびその特異的な遺伝子発現阻害のための方法 |
| US10913767B2 (en) | 2010-04-22 | 2021-02-09 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising acyclic and abasic nucleosides and analogs |
| US20130190383A1 (en) | 2010-04-26 | 2013-07-25 | Marina Biotech, Inc. | Nucleic acid compounds with conformationally restricted monomers and uses thereof |
| WO2015106128A2 (en) * | 2014-01-09 | 2015-07-16 | Alnylam Pharmaceuticals, Inc. | MODIFIED RNAi AGENTS |
| IL316808A (en) | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| JP7105065B2 (ja) * | 2014-12-15 | 2022-07-22 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | リガンド修飾二本鎖核酸 |
| WO2016209862A1 (en) | 2015-06-23 | 2016-12-29 | Alnylam Pharmaceuticals, Inc. | Glucokinase (gck) irna compositions and methods of use thereof |
| JP6924744B2 (ja) | 2015-07-17 | 2021-08-25 | アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | Ｂ型肝炎ウイルスに対する組成物および薬剤ならびにその使用 |
| KR102645243B1 (ko) * | 2016-11-23 | 2024-03-11 | 알닐람 파마슈티칼스 인코포레이티드 | 표적외 효과가 감소된 변형 rna 작용제 |
-
2019
- 2019-05-16 WO PCT/US2019/032633 patent/WO2019222479A1/en not_active Ceased
- 2019-05-16 JP JP2020564156A patent/JP7384833B2/ja active Active
- 2019-05-16 AU AU2019270152A patent/AU2019270152B2/en active Active
- 2019-05-16 CA CA3099930A patent/CA3099930A1/en active Pending
- 2019-05-16 EP EP19803000.9A patent/EP3802827A4/en active Pending
- 2019-05-16 US US17/055,710 patent/US12247203B2/en active Active
-
2022
- 2022-05-13 US US17/743,663 patent/US12275938B2/en active Active
-
2023
- 2023-11-07 JP JP2023189998A patent/JP7684373B2/ja active Active
-
2024
- 2024-12-04 US US18/968,749 patent/US20250207131A1/en active Pending
-
2025
- 2025-05-01 JP JP2025076285A patent/JP2025113272A/ja active Pending
- 2025-07-22 AU AU2025206406A patent/AU2025206406A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2019270152A1 (en) | 2020-12-10 |
| US20250207131A1 (en) | 2025-06-26 |
| JP7384833B2 (ja) | 2023-11-21 |
| JP7684373B2 (ja) | 2025-05-27 |
| US12247203B2 (en) | 2025-03-11 |
| AU2025206406A1 (en) | 2025-08-14 |
| EP3802827A4 (en) | 2022-08-03 |
| US20210238595A1 (en) | 2021-08-05 |
| JP2025113272A (ja) | 2025-08-01 |
| US12275938B2 (en) | 2025-04-15 |
| JP2021523720A (ja) | 2021-09-09 |
| JP2024014905A (ja) | 2024-02-01 |
| EP3802827A1 (en) | 2021-04-14 |
| AU2019270152B2 (en) | 2025-05-08 |
| US20220290145A1 (en) | 2022-09-15 |
| WO2019222479A1 (en) | 2019-11-21 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20240516 |
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| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT Effective date: 20241217 |
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| W00 | Other event occurred |
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Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT Effective date: 20250318 |
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| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD Year of fee payment: 6 |
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| U00 | Fee paid |
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| D15 | Examination report completed |
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| P13 | Application amended |
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